Other antianemic preparations

• What is imetelstat?
• How does imetelstat work?
• Which conditions is imetelstat being studied for?
• How is imetelstat given?
• What do the clinical trials show?
• What side effects are known?

What is imetelstat?

Imetelstat is an investigational substance being studied for the treatment of myelofibrosis, a type of blood cancer in which scar tissue builds up in the bone marrow and disrupts normal blood cell production.^[1] The trial record lists it as a drug given by intravenous infusion at a dose of 9.4 mg per kilogram of body weight.^[1] No marketing authorisation information is provided for imetelstat in these records, which suggests it is still under investigation.

How does imetelstat work?

The trial record does not describe the mechanism of action of imetelstat. The available sources focus on other antianemic preparations, such as oral and intravenous iron products, rather than on imetelstat itself.

Which conditions is imetelstat being studied for?

• Myelofibrosis — a rare blood cancer in which the bone marrow is replaced by scar tissue, leading to low blood counts and an enlarged spleen. The trial focuses on intermediate-2 or high-risk disease that has relapsed or is refractory to JAK-inhibitor treatment.^[1]

How is imetelstat given?

Imetelstat is given by intravenous infusion at a dose of 9.4 mg per kilogram of body weight.^[1] The trial record does not specify the frequency of administration or the duration of treatment.

What do the clinical trials show?

One Phase 3 trial is authorised. It is an interventional study comparing imetelstat with best available therapy in 354 participants with intermediate-2 or high-risk myelofibrosis whose disease is relapsed or refractory to JAK-inhibitor treatment.^[1] The primary outcome is overall survival, defined as the time interval from randomisation date to date of death from any cause.^[1]

What side effects are known?

The supplementary sources provided relate to iron-containing antianemic preparations, not to imetelstat. They describe hypersensitivity reactions, including anaphylactic reactions, associated with intravenous iron products such as ferric carboxymaltose, iron sucrose, iron dextran and iron gluconate.^[3][2] These side effects do not apply to imetelstat and are mentioned only because they appear in the sources for other antianemic preparations. No side effect information specific to imetelstat is available in the data provided.